A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 in Patients With Friedreich's Ataxia

The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.

Study Overview

• Status: Recruiting
• Conditions: Friedreich Ataxia
• Intervention / Treatment: Drug: DT-216P2

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Varlli Scott; Phone Number: +613 8341 6228; Email: Varlli.scott@mcri.edu.au

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2031
      • Recruiting
      • Scientia Clinical Research Ltd
      • Contact: Varlli Scott; Phone Number: +613 8341 6228; Email: Varlli.scott@mcri.edu.au
  • Victoria
    • East Melbourne, Victoria, Australia, 3002
      • Recruiting
      • Doherty Clinical Trials
      • Contact: Varlli Scott; Phone Number: +613 8341 6228; Email: Varlli.scott@mcri.edu.au
    • Melbourne, Victoria, Australia, 3004
      • Recruiting
      • Nucleus Network
      • Contact: Varlli Scott; Phone Number: +613 8341 6228; Email: Varlli.scott@mcri.edu.au

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participants must be 18-65 years of age inclusive, at the time of signing the informed consent.
• Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansions in the frataxin gene.
• Stage 5.5 or less on the FSA at screening.
• BMI between 16 and 32 kg/m2 at screening; weight should be <= 100 kg at screening.
• Male and/or female using protocol defined and regulatory approved contraception.
• Capable of giving signed informed consent.

Exclusion Criteria:

• Any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study protocol.
• Any clinically significant nonmedical conditions and psychiatric disorders that could put the participant at higher risk for participation in the study, influence the participant's ability to participate in the study, or interfere with interpretation of the participant's study results, in the opinion of the investigator.
• Received an investigational agent within the last 30 days or 5 half-lives, whichever longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to study enrollment. Exception: Potential participants who are currently on Omaveloxolone must be on stable doses for at least 3 months.
• Is not willing to comply with the contraceptive requirements during the study period, as per protocol.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: DT-216P2	Drug: DT-216P2; Active

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Frequency of treatment-emergent adverse events (TEAE)	Up to Week 12

Secondary Outcome Measures

Outcome Measure	Time Frame
Area under the curve (AUClast, AUCinf)	Up to Week 12
Maximum concentration (Cmax)	Up to Week 12
Time to Cmax (Tmax)	Up to Week 12
Half-life (t1/2)	Up to Week 12
Frataxin expression at baseline and after treatment will be measured	Up to Week 12

Collaborators and Investigators

• Sponsor: Design Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): March 1, 2027

Study Registration Dates

• First Submitted: February 25, 2025
• First Submitted That Met QC Criteria: March 11, 2025
• First Posted (Actual): March 13, 2025

Study Record Updates

• Last Update Posted (Actual): May 18, 2025
• Last Update Submitted That Met QC Criteria: May 14, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolic Diseases; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Spinal Cord Diseases; Dyskinesias; Mitochondrial Diseases; Cerebellar Diseases; Spinocerebellar Degenerations; Ataxia; Cerebellar Ataxia; Friedreich Ataxia
• Other Study ID Numbers: DTX-216P2-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No